Ultragenyx's Hypophosphatemia Drug Succeeds In Phase III

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and its Japanese partner Kyowa Hakko Kirin Co., Ltd announced positive data from a phase III study evaluating its pipeline candidate burosumab (KRN23) in adults with X-linked hypophosphatemia (XLH).

The 24-week study met the primary endpoint as the patients treated with burosumab achieved a statistically significant improvement in serum phosphorus levels as, 94% of patients achieved normal levels compared with 8% on placebo. Further, the secondary endpoint of the study were also met as patients treated with burosumab achieved significant improvement in stiffness and witnessed strong improvements in physical function and pain.

Shares of the company increased 13.2% following the news release. However, Ultragenyx’s share price movement shows that the stock has underperformed the Zacks classified Medical-Biomedical/Genetics industry year to date. Specifically, the stock is up 1.3% compared with the industry’s gain of 2.9%.

Moving ahead, the company plans to make regulatory filings in U.S. and Europe, based on the above data.The latest news comes on the heels of positive 64-week data, announced last week, from a pediatric phase II study, evaluating burosumab for the treatment of XLH in children aged between five and twelve years. The multicentre, open-label, dose-finding phase II study (n=52) also showed improvement in serum phosphorus levels, rickets, growth rates and other functional outcomes with use of burosumab, sustained through 64 weeks of treatment.

In fact, in Jun 2016 the FDA had granted breakthrough therapy designation to burosumab for the treatment of XLH in pediatric patients aged one year and above.

Currently, burosumab is also being developed in a phase II study for the treatment of tumor-induced osteomalacia (TIO).

We note that Ultragenyx is making impressive progress with its pipeline candidates. The company expects to submit regulatory filings for rhGUS (UX003) for the treatment of mucopolysaccharidosis 7 (MPS 7) in both the U.S. and the EU in the first half of 2017.

Going forward, we expect investor’s focus to remain on the Committee for Medicinal Products for Human Use’s (CHMP) opinion on KRN23 and the company’s regulatory filing for rhGUS.

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the health care sector include Heska Corporation (NASDAQ:HSKA) , Galena Biopharma, Inc. (NASDAQ:GALE) and Infinity Pharmaceuticals, Inc. (NASDAQ:INFI) . Each of these stock sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Heska’s earnings per share estimates increased from $1.53 to $1.65 for 2017 and from $1.90 to $2.01 for 2018, over the last 60 days. The company posted positive surprises in three of the four trailing quarters with an average beat of 291.54%. Its share price increased 41.9% year to date.

Galena’s loss per share estimates narrowed from $2.27 to 58 cents for 2017 and from 92 cents to 73 cents for 2018, over the last 60 days. The company posted positive earnings surprises in two of the four trailing quarters, with an average beat of 53.83%.

Infinity Pharma’s loss per share estimates narrowed from $1.10 to $1.03 for 2017 over the last 60 days. The company posted positive earnings surprises in three of the four trailing quarters, with an average beat of 36.64%.

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